Queen's University Cancer Research Institute, Kingston, Canada.
Clin Cancer Res. 2010 Dec 15;16(24):5963-71. doi: 10.1158/1078-0432.CCR-10-1962.
In the era of molecular oncology, patients still define a useful therapy as one that allows them to live longer and helps them to live better. Although patient outcomes have clearly improved as a result of randomized controlled trials (RCT), it is critical that contemporary trials retain the perspective of these fundamental patient-centered outcomes. Trends in study design, results, and interpretation of oncology RCTs from the past provide a useful framework in which to consider how the research community may approach trial design in the future. Although the RCT remains the standard for establishing efficacy, this article also considers how population-based outcome studies can provide insight into effectiveness of new therapies and explores how the results of RCTs translate into benefit in the general population.
在分子肿瘤学时代，患者仍然将能够延长生存期和提高生活质量的治疗方法定义为有效治疗。虽然随机对照试验（RCT）的结果明显改善了患者的预后，但重要的是，当代试验应保留这些以患者为中心的基本结局的观点。回顾过去肿瘤学 RCT 的研究设计、结果和解释趋势为我们提供了一个有用的框架，使我们可以思考研究界将来如何进行试验设计。虽然 RCT 仍然是确定疗效的标准，但本文还考虑了基于人群的结局研究如何为新疗法的有效性提供见解，并探讨了 RCT 结果如何转化为普通人群的获益。
